2021
DOI: 10.1021/acs.chemrev.0c00997
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Polymeric Delivery of Therapeutic Nucleic Acids

Abstract: The advent of genome editing has transformed the therapeutic landscape for several debilitating diseases, and the clinical outlook for gene therapeutics has never been more promising. The therapeutic potential of nucleic acids has been limited by a reliance on engineered viral vectors for delivery. Chemically defined polymers can remediate technological, regulatory, and clinical challenges associated with viral modes of gene delivery. Because of their scalability, versatility, and exquisite tunability, polymer… Show more

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Cited by 210 publications
(296 citation statements)
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“… 9 Viral delivery is confronted with logistical, technological, and commercial obstacles in the form of limited cargo capacity, 10 high manufacturing costs, 11 significant regulatory burdens, 12 and severe immune responses. 13 − 15 To circumvent these challenges, biomaterials researchers have designed chemically defined synthetic delivery platforms such as polymers 16 and lipids 17 whose performance meets or exceeds benchmarks set by clinically deployed viral vectors. 18 , 19 …”
Section: Introductionmentioning
confidence: 99%
“… 9 Viral delivery is confronted with logistical, technological, and commercial obstacles in the form of limited cargo capacity, 10 high manufacturing costs, 11 significant regulatory burdens, 12 and severe immune responses. 13 − 15 To circumvent these challenges, biomaterials researchers have designed chemically defined synthetic delivery platforms such as polymers 16 and lipids 17 whose performance meets or exceeds benchmarks set by clinically deployed viral vectors. 18 , 19 …”
Section: Introductionmentioning
confidence: 99%
“…However, the clinical implementation of mRNA therapeutics is limited by their poor bioavailability and instability in vivo [7][8][9]. To address these obstacles and improve the druggability of mRNA, tremendous efforts have been invested in engineering mRNA molecules and developing mRNA delivery systems [10][11][12][13]. Lipids and polymers are the most advanced delivery systems for nucleic acid therapeutics [14][15][16].…”
Section: Introductionmentioning
confidence: 99%
“…The therapeutic effect of gene therapy largely depends on the capacity of gene vectors to deliver exogenous genes and the efficiency of gene transfection in target cells [ 4 ]. Most of the related work focused on optimizing gene delivery systems to enhance the efficiency of gene therapy through effective encapsulation, enhanced cellular uptake of target cells, endosomal escape and promoted nuclear transport [ 5 , 6 ]. These methods only achieved highly efficient gene transfection in vitro, the therapeutic effect in vivo was still far from satisfactory [ 7 ].…”
Section: Introductionmentioning
confidence: 99%